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Trial · Interventional

TerminatedPhase 2/3Results postedRegistry data

Clinical Trial of Growth Hormone in MPS I, II, and VI

NCTNCT00748969 (opens ClinicalTrials.gov in a new tab)Other id0808M43681
Open at ClinicalTrials.govAsk about this trial

Overview

Status
Terminated
Phase
Phase 2/3
Enrollment
2 participants (actual)
Sponsor
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center · Other
Design
Randomized · Open label (no masking)
Countries
1 country

United States

Start
Nov 2008
Primary completion
Sept 2013
Completion
Sept 2013
First posted
9 Sept 2008 (estimated)
Last update posted
5 Sept 2018
Conditions
Mucopolysaccharidosis I · Mucopolysaccharidosis II · Mucopolysaccharidosis VI

Timeline

1 trial · 1 stopped early · 1 with posted results

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
2008200920102011201220132014
  1. Ph 2/3

    Clinical Trial of Growth Hormone in MPS I, II, and VI

    NCT00748969

    Nov 2008 to Sept 2013

    Terminated

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

The purpose of this study is to determine whether growth hormone is a safe and effective treatment for short stature in children with Mucopolysaccharidosis type I, II, and VI.

Interventions

  • Somatropin (DNA origin)Drug

Arms2

  • Experimental

    Growth hormone treatmen

    Growth hormone treatment arm. Somatropin (DNA origin)

  • No intervention

    No growth hormone treatment in year 1

    No growth hormone treatment in year 1; option for treatment in year 2 open-label period.

Primary outcomes1

Outcomes the sponsor listed as primary.

  1. Change in Growth Velocity From Baseline to End of Study Year 1.

    Time frame · 12 months

Secondary outcomes(1)

  1. Safety: Number Drug Related SAEs

    Time frame · 1 months

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeNumber· 12 months

Change in Growth Velocity From Baseline to End of Study Year 1.

GroupValue (cm/yr)
Growth Hormone TreatmentNA
No Growth Hormone Treatmentnot posted
Secondary and other outcome results(1)
Secondary outcomeCount of participants· 1 months

Safety: Number Drug Related SAEs

GroupValue (Participants)
Growth Hormone Treatment0
No Growth Hormone Treatment0

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
GroupDeathsSerious eventsOther events
GH TreatmentGrowth hormone treatment arm. Somatropin (DNA origin) Somatropin (DNA origin): The study starting dose of Nutropin AQ® will be 0.48 mg/kg/week divided into daily SC injections. Nutropin AQ® will be administered by either the subject or, if unable to demonstrate competency in this, then by the guardian. To decrease the risk of increased intracranial hypertension, the dose in the first month of treatment will be decreased by 50% (0.24 mg/kg/week), and then increased to 0.48 mg/kg/week if tolerated well after 1 month.–0 / 11 / 1
No GH TreatmentNo placebo/no treatment–0 / 00 / 0

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Eligibility

Who could join, as posted.

Sex
All
Age
from 5 Years up to 17 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
Inclusion Criteria: * A parent or legally authorized representative must provide written informed consent and comply with study assessments for the full duration of the study. * Chronologic age ≥ 5 years and bone age ≤12 years * Diagnosis of MPS I, II, or VI * Height ≤ -2 SDS for age and gender * Ability to travel to study center for evaluations. * Ability of the participant to cooperate with study procedures, to notify a guardian of symptoms, and provide assent for participation in the study. Exclusion Criteria: * History of treatment with hGH * Untreated pituitary deficiency * Pregnancy (positive urine pregnancy test) prior to enrollment in the study * Participation in another simultaneous medical intervention trial * Patients with closed epiphysis * Active neoplasm * Orthopedic procedure of the femur within the last 6 months. * Known or suspected allergy to trial product or related products. * Structural lesion on brain MRI resulting in brain compression * Any other social or medical condition that the investigator believes would pose a significant hazard to the subject if the investigational therapy were initiated or be detrimental to the study. * Obstructive sleep apnea without BiPAP or tonsillectomy/adenoidectomy treatment. * CNS shunt. * Abnormal cardiac function based on echocardiogram within 6 months prior to enrollment : * Ejection fraction less than 50% * Left ventricular chamber size greater than or less than 2 standard deviations of normal for body surface area * Left ventricular wall thickness greater than or less than 2 standard deviations of normal for body surface area * More than mild to moderate aortic insufficiency with abdominal aortic run-off * More than mild to moderate mitral insufficiency with pulmonary hypertension * Abnormal pulmonary function based on pulmonary function tests within 6 months prior to enrollment: * abnormal FVC \< 80% of predicted for age, gender, and height * abnormal FEV1 \< 80% predicted for age, gender, and height * abnormal FEV1/FVC * abnormal oxygen saturation

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